New CRISPR Tool May Help Prevent Worst Genetic Diseases

The CRISPR gene editing technology has been popularly used together with Cas9, a protein that works like a snipping tool. Another protein, the CPf1, is now showing greater promise in editing human cells because of its smaller size and simplicity.

Scientists from the Univerity of Texas reported in Science Advances that CRISPR-Cpf1 can be used to correct mutations associated with Duchenne muscular dystrophy, a disease that leads to muscular degeneration. They used the CRISPR-Cpf1 in human heart muscle cells and successfully prevented the progress of the disease. In mice with such disease, the genetic tool successfully reversed an inflammatory symptom.

The researchers stressed that CRISPR-Cpf1 could be a powerful tool in finding a cure for human diseases linked to various mutations.

Read more in Science Advances.